Arrowhead Pharmaceuticals, Inc. has announced new interim clinical findings on ARO-RAGE, an RNAi-based therapy designed to treat inflammatory lung conditions such as asthma, during the American Thoracic Society (ATS) 2024 International Conference. The ongoing Phase 1/2 study showed promising results, indicating that ARO-RAGE treatment led to a reduction in soluble RAGE (sRAGE) concentration in bronchoalveolar lavage fluid (BALF) and serum, demonstrating a dose-dependent effect in both normal healthy volunteers (NHV) and patients with mild to moderate asthma.

James Hamilton, M.D., Chief of Discovery and Translational Medicine at Arrowhead, emphasized the significance of these results, highlighting the potential of their proprietary TRiMTM platform in developing impactful therapies for various pulmonary diseases. He stressed that Arrowhead was the first to demonstrate high levels of target gene knockdown in the lung in healthy humans using RNAi, and these new data further validate their approach.

Key ARO-RAGE Results from the ongoing 1001 study as of April 5, 2024, include:

• Dose-dependent reductions in sRAGE observed in both BALF and serum of NHVs after single and multiple doses of ARO-RAGE.
• In patients with mild to moderate asthma, serum sRAGE reduction of up to 88% was achieved after two doses, with a mean maximum reduction of up to 77%.
• Similar dose-responsive reductions in serum sRAGE were observed in NHVs and asthma patients at each dose level.
• Long-lasting pharmacodynamic effects support the potential for once-every-two-month dosing.
• Limited and short-lived ARO-RAGE plasma exposure in NHVs following a single dose.

Safety and tolerability results indicated a favorable profile with no notable impact on systemic safety labs or lung function over time, and no serious adverse effects related to the study drug.

Additionally, Arrowhead presented preclinical data on two other lung-targeted programs, ARO-TSLP and ARO-IAV, which utilize their TRiMTM platform. ARO-TSLP targets thymic stromal lymphopoietin (TSLP), while ARO-IAV aims to silence highly conserved influenza A viruses, including H5N1.

The presentations at the ATS 2024 International Conference provided further insight into these developments. ARO-RAGE's potential as a treatment for inflammatory lung diseases stems from its ability to target the receptor for advanced glycation end-products (RAGE), a key mediator implicated in asthma and other inflammatory conditions.

The ARORAGE-1001 Phase 1/2 study (NCT05276570) continues to assess the safety, pharmacokinetics, and pharmacodynamics of ARO-RAGE in NHVs and patients with asthma, showcasing promising results that support further clinical advancement.

 

Source: businesswire.com

Report Abuse